FDA changes rare disease tune again as it clears path for Regenxbio’s gene therapy - Pharmaceutical Technology

With a regulatory route now in place, Regenxbio expects to meet with the FDA again in July, with a BLA resubmission to follow in Q3 2026. With a regulatory route now in place, Regenxbio expects to meet with the FDA again in July, with a BLA resubmission to follow in Q3 2026. Regenxbio’s positive announcement comes as the FDA is showing less rigidity in the rare disease drug development space. Credit: Teacher Photo/Shutterstock.com. The US Food and Drug Administration (FDA) will reconsider Regenxbio’s gene therapy for Hunter syndrome after determining the preexisting package adequate for a review, marking the latest…

Source: Pharmaceutical Technology